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Study to evaluate the efficacy and safety of MAS825 in patients with autoinflammatory diseases.
EU CTIS ID: 2023-504419-34-00
What this study is testing
To determine the efficacy of MAS825 in prevention of flares in patients with autoinflammatory diseases, including NLRC4-GOF, XIAP deficiency, or CDC42 mutations.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- All cohorts: 1.Male and female patients weighing at least 3 kg
- All cohorts: 2.Written informed consent by parent(s)/legal guardian(s) for the pediatric patients and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed. For adult patients, written informed consent by patients capable of giving consent, or, when the patient is not capable of giving consent, by his or her legal/authorized representative (if allowed according to local requirements).
- Cohort 1: 3.Patients with a genetic diagnosis of either NLRC4-GOF, XIAP deficiency, or CDC42 mutation.
- Cohort 1: 4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42.
- Cohort 1: 5.At first treatment, evidence of active disease
- Cohort 2: 6.Patients with a genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutations who are being treated with MAS825 in a Novartis managed access program.
You likely can't join if
- 1.History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.
- 10.Patients weighing >160 kg at Screening.
- 11. For CDC42 mutation patients: Takenouchi-Kosaki syndrome – CDC42 mutations associated with a diverse syndrome characterized by variable development delays, cardiac, brain and hematological abnormalities.
- 2.Signs and symptoms, in the judgment of the investigator, of clinically significant systemic recurrent and/or evidence of active bacterial, fungal, parasitic or viral infections, excluding chronic Epstein-Barr Virus (EBV).
- 3.Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for MAS825 therapy
- 4.Previous treatment with anti-rejection and/or immunomodulatory drugs within the past 28 days or 5 half-lives (whichever is the longer) for immunomodulatory therapeutic antibodies prior to MAS825 treatment with the exceptions of: - glucocorticoids - cyclosporin - targeted binding or blocking therapies, which must be discontinued prior to treatment with MAS825 - drugs listed as prohibited medication in the protocol, for which the washout periods should be followed as outlined in the protocol.
See the full eligibility criteria
Who can join
- All cohorts: 1.Male and female patients weighing at least 3 kg
- All cohorts: 2.Written informed consent by parent(s)/legal guardian(s) for the pediatric patients and assent by the pediatric patient (depending on local requirements) must be obtained before any study-specific assessment is performed. For adult patients, written informed consent by patients capable of giving consent, or, when the patient is not capable of giving consent, by his or her legal/authorized representative (if allowed according to local requirements).
- Cohort 1: 3.Patients with a genetic diagnosis of either NLRC4-GOF, XIAP deficiency, or CDC42 mutation.
- Cohort 1: 4.Clinical history and investigations consistent with autoinflammation with infantile enterocolitis (AIFEC/NLRC4-GOF), XIAP or CDC42.
- Cohort 1: 5.At first treatment, evidence of active disease
- Cohort 2: 6.Patients with a genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutations who are being treated with MAS825 in a Novartis managed access program.
What rules you out
- 1.History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes or to any of the excipients.
- 10.Patients weighing >160 kg at Screening.
- 11. For CDC42 mutation patients: Takenouchi-Kosaki syndrome – CDC42 mutations associated with a diverse syndrome characterized by variable development delays, cardiac, brain and hematological abnormalities.
- 2.Signs and symptoms, in the judgment of the investigator, of clinically significant systemic recurrent and/or evidence of active bacterial, fungal, parasitic or viral infections, excluding chronic Epstein-Barr Virus (EBV).
- 3.Any conditions or significant medical problems, which in the opinion of the investigator places the patient at unacceptable risk for MAS825 therapy
- 4.Previous treatment with anti-rejection and/or immunomodulatory drugs within the past 28 days or 5 half-lives (whichever is the longer) for immunomodulatory therapeutic antibodies prior to MAS825 treatment with the exceptions of: - glucocorticoids - cyclosporin - targeted binding or blocking therapies, which must be discontinued prior to treatment with MAS825 - drugs listed as prohibited medication in the protocol, for which the washout periods should be followed as outlined in the protocol.
- 5.A positive HIV test result at Screening. Evidence of prior testing within 3 months is sufficient.
- 6.A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C test result. Evidence of prior testing within 3 months is sufficient.
- 7.Presence of tuberculosis infection as defined by a positive TB test at Screening. Evidence of prior testing within 3 months is sufficient.
- 8.Live vaccinations within 1 month prior to MAS825 treatment, during the trial, and up to 3 months following the last dose.
- 9.Female patients of child-bearing potential (or Tanner stage 2 or above) who are or might become sexually active, agree to use highly effective contraceptive methods to prevent pregnancy while on MAS825 therapy.
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- Canada
- Japan
- United Kingdom
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; Canada; Japan; United Kingdom; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.